ATE437944T1 - DNA SEQUENCES CODING FOR DYSTROPHIN MINIGEN AND METHOD FOR USE THEREOF - Google Patents

DNA SEQUENCES CODING FOR DYSTROPHIN MINIGEN AND METHOD FOR USE THEREOF

Info

Publication number
ATE437944T1
ATE437944T1 AT01934920T AT01934920T ATE437944T1 AT E437944 T1 ATE437944 T1 AT E437944T1 AT 01934920 T AT01934920 T AT 01934920T AT 01934920 T AT01934920 T AT 01934920T AT E437944 T1 ATE437944 T1 AT E437944T1
Authority
AT
Austria
Prior art keywords
dystrophin
present
provides
minigen
dna sequences
Prior art date
Application number
AT01934920T
Other languages
German (de)
Inventor
Xiao Xiao
Original Assignee
Asklepios Biopharmaceutical In
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Asklepios Biopharmaceutical In filed Critical Asklepios Biopharmaceutical In
Application granted granted Critical
Publication of ATE437944T1 publication Critical patent/ATE437944T1/en

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Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • C07K14/4701—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals not used
    • C07K14/4707—Muscular dystrophy
    • C07K14/4708—Duchenne dystrophy
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2799/00—Uses of viruses
    • C12N2799/02—Uses of viruses as vector
    • C12N2799/021—Uses of viruses as vector for the expression of a heterologous nucleic acid
    • C12N2799/025—Uses of viruses as vector for the expression of a heterologous nucleic acid where the vector is derived from a parvovirus

Abstract

The present invention provides a series of novel dystrophin minigenes that retain the essential biological functions. The expression of the dystrophin minigenes may be controlled by a regulatory element along with a small polyadenylation signal. The entire gene expression cassettes may be readily packaged into a viral vector, preferably an AAV vector. The present invention further defines the minimal functional domains of dystrophin and provides ways to optimize and create new versions of dystrophin minigenes. Finally, the present invention provides a method of treatment for Duchenne muscular dystrophy (DMD) and Becker muscular dystrophy (BMD).
AT01934920T 2000-04-28 2001-04-27 DNA SEQUENCES CODING FOR DYSTROPHIN MINIGEN AND METHOD FOR USE THEREOF ATE437944T1 (en)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US20077700P 2000-04-28 2000-04-28
PCT/US2001/013677 WO2001083695A2 (en) 2000-04-28 2001-04-27 Dna sequences encoding dystrophin minigenes and methods of use thereof

Publications (1)

Publication Number Publication Date
ATE437944T1 true ATE437944T1 (en) 2009-08-15

Family

ID=22743139

Family Applications (1)

Application Number Title Priority Date Filing Date
AT01934920T ATE437944T1 (en) 2000-04-28 2001-04-27 DNA SEQUENCES CODING FOR DYSTROPHIN MINIGEN AND METHOD FOR USE THEREOF

Country Status (9)

Country Link
US (2) US7001761B2 (en)
EP (1) EP1287125B1 (en)
CN (1) CN100422320C (en)
AT (1) ATE437944T1 (en)
AU (1) AU2001261063A1 (en)
CA (1) CA2407309C (en)
DE (1) DE60139394D1 (en)
ES (1) ES2330615T3 (en)
WO (1) WO2001083695A2 (en)

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WO2019204303A2 (en) * 2018-04-16 2019-10-24 The Trustees Of The University Of Pennsylvania Compositions and methods for treating duchenne muscular dystrophy
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Also Published As

Publication number Publication date
WO2001083695A9 (en) 2002-12-12
WO2001083695A3 (en) 2002-03-07
WO2001083695A2 (en) 2001-11-08
ES2330615T3 (en) 2009-12-14
AU2001261063A1 (en) 2001-11-12
EP1287125A2 (en) 2003-03-05
CA2407309A1 (en) 2001-11-08
EP1287125B1 (en) 2009-07-29
CN100422320C (en) 2008-10-01
US20060073586A1 (en) 2006-04-06
CN1439051A (en) 2003-08-27
US7001761B2 (en) 2006-02-21
US20030171312A1 (en) 2003-09-11
EP1287125A4 (en) 2005-04-27
DE60139394D1 (en) 2009-09-10
CA2407309C (en) 2011-08-02
US7510867B2 (en) 2009-03-31

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